A firm making gene therapy for rare diseases that was co-founded by a UMass Medical School professor has won regulatory approval to continue research and investigatory work for an ALS therapy.
Apic Bio, now based in Cambridge’s Kendall Square, was co-founded by Dr. Robert Brown Jr., a professor in UMass’ neurology department. Brown was a lead member of the team that discovered a particular enzyme as the first genetic mutation linked to ALS, or amyotrophic lateral sclerosis.
[Related:Â UMass Medical School announces ALS breakthrough]
The new therapy whose investigational new drug application was approved by the U.S. Food and Drug Administration began nearly 30 years ago with the gene therapy research conducted by Brown and others. The therapy, whose approval Apic Bio announced April 21, is aimed at slowing or reversing the progression of ALS, a disease also known as Lou Gehrig’s disease with no cure and whose cause is unknown.
First and second phase clinical trials are planned for late this year or early next year to evaluate the safety and effectiveness of the therapy in patients.
